Induced Pluripotent Stem Cells to Understand Mucopolysaccharidosis. I: Demonstration of a Migration Defect in Neural Precursors

Induced Pluripotent Stem Cells to Understand Mucopolysaccharidosis. I: Demonstration of a Migration Defect in Neural Precursors

<i>Background</i>: Mucopolysaccharidosis type I-Hurler (MPS1-H) is a severe genetic lysosomal storage disorder due to loss-of-function mutations in the <i>IDUA</i> gene. The subsequent complete deficiency of alpha l-iduronidase enzyme is directly responsible of a progressive...

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Bibliographic Details
Main Authors: Silvin Lito, Adama Sidibe, Sten Ilmjarv, Patricie Burda, Matthias Baumgartner, Bernhard Wehrle-Haller, Karl-Heinz Krause, Antoine Marteyn
Format: Article
Language:English
Published: MDPI AG 2020-12-01
Series:Cells
Subjects:
induced pluripotent stem cells
neuronal differentiation
disease modelling
mucopolysaccharidosis I
neural migration
neurite outgrowth
Online Access:https://www.mdpi.com/2073-4409/9/12/2593

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https://www.mdpi.com/2073-4409/9/12/2593

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